The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy ...
Columnist Patrick Moeschen shares what he wishes doctors understood about treating patients with rare diseases such as ...
Patients aged 10-50 are being sought for a clinical trial testing SAR446268, a gene therapy for myotonic dystrophy type 1 ...
I was diagnosed with Duchenne muscular dystrophy at age 3. By the time I was 10, I was exclusively using a power wheelchair for mobility — and I’ve been rolling ever since. Living with a chronic ...
A combined regimen of testosterone and growth hormone led to significant improvements in muscle mass, strength, and mobility in men with facioscapulohumeral muscular dystrophy (FSHD) in a clinical ...
Living with Duchenne muscular dystrophy means every trip to the hospital is key to my survival. From heart scans to routine medical appointments, leaving home is never simple. I rely on my mom to ...
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback